{"id":"W4416431787","doi":"10.1016/j.mam.2025.101424","title":"Precision gene therapy: Tailoring rAAV-mediated gene therapies for inherited retinal dystrophies (IRDs)","year":2025,"lang":"en","type":"review","venue":"Molecular Aspects of Medicine","topic":"Retinal Development and Disorders","field":"Biochemistry, Genetics and Molecular Biology","cited_by":1,"is_retracted":false,"has_abstract":true,"ca_institutions":"","funders":"Novartis Stiftung für Medizinisch-Biologische Forschung; Foundation Fighting Blindness","keywords":"Genetic enhancement; Retinal pigment epithelium; Gene delivery; Retinal; Gene; Retina; Genome editing; Transgene; Retinal ganglion cell","routes":{"ca_aff":false,"ca_fund":true,"ca_venue":false,"about_ca":false,"invisible_to_affiliation_only":true},"retraction":null,"screen":null,"direct_labels":[],"prediction":{"model_version":"metacan-v3-hybrid-931329e0061c","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.0003805399,0.0006806737,0.0008067318,0.001507564,0.0001640446,0.0006713316,0.0006784526,0.0008474547,0.002225046],"category_scores_gemma":[0.0003603529,0.0002102562,0.0003984772,0.001134374,0.0002861202,0.0007311131,0.0004805741,0.001376871,0.001343631],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0004309866,"about_ca_system_score_gemma":0.0005399938,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0006076706,"about_ca_topic_score_gemma":0.001092681,"domain_scores_codex":[0.9998713,0.00001711413,0.00001392107,0.00002292348,0.0000584923,0.00001617193],"domain_scores_gemma":[0.9998928,0.00004956696,0.00001625919,0.000003740224,0.00002549397,0.00001216736],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"design_other","study_design_gemma":"not_applicable","study_design_scores_codex":[0.00006113166,0.0000753217,0.0001046949,0.01114784,0.00007438747,0.0002312702,0.00005658922,0.0005579588,0.008270704,0.004788785,0.01721447,0.9574169],"study_design_scores_gemma":[0.00001867812,0.0001211463,0.0004604539,0.001416811,0.00007776913,0.001157933,0.00003090818,0.0001401001,0.002140227,0.001349321,0.9930713,0.00001543764],"study_design_candidate":"not_applicable","study_design_consensus":null,"genre_codex":"review","genre_gemma":"review","genre_scores_codex":[0.000355986,0.9969319,0.0004645189,0.0001746532,0.0002135002,0.000008927694,0.00002401016,0.00001861572,0.001807923],"genre_scores_gemma":[0.001169539,0.9971597,0.0004092789,0.0001685795,0.00006882937,0.00001131545,0.000045328,0.00000289385,0.0009645419],"genre_candidate":"review","genre_consensus":"review","teacher_disagreement_score":0.002225046,"threshold_uncertainty_score":0.007443488,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.02051490841312063,"score_gpt":0.3027497237540689,"score_spread":0.2822348153409482,"validation_status":"score_only:v0-immature-baseline","note":"Baseline scores from an immature model (maturity gate not passed). Scores rank; they never assert a category."}}